# STARFiSH: Study of Testosterone and rHGH in FSHD: A Proof-of-Concept Study

> **NIH NIH R01** · UNIVERSITY OF ROCHESTER · 2020 · $471,791

## Abstract

Facioscapulohumeral muscular dystrophy (FSHD) is the second most common adult muscular dystrophy in the
world with a global prevalence of ~4:100,000. Clinically, patients with FSHD experience progressive
weakness, muscle wasting, fatigue, and respiratory decline. As adult patients with FSHD age, they frequently
develop difficulty walking or lose the ability to ambulate due to profound weakness and muscle atrophy.
Currently, there are no therapies that have been demonstrated to reverse or even slow the progressive
symptoms associated with FSHD. Large scale clinical trials have found that testosterone combined with
recombinant human growth hormone (rHGH) (combination therapy) is well tolerated and effective in
synergistically improving respiratory function, lean body mass, protein synthesis, strength, and aerobic
endurance in healthy adult human populations. Both testosterone and rHGH are readily available and
approved for human use but have never been formally studied together in a muscular dystrophy population.
We propose a 36-week, proof-of-concept clinical study of the safety and tolerability of daily rHGH combined
with biweekly testosterone injections in men with FSHD. All participants will be serially and closely monitored
during a 24 week period of combination therapy followed by a 12 week washout period. Safety assessments
will include monitoring for medication side effects, laboratory abnormalities, physical exam changes, and EKG
alterations. As a secondary objective, we will examine the pharmacokinetic effects of combination therapy on
lean body mass and serum biomarkers. Participants will also have serial assessments of their ambulation,
strength, physical function, patient-reported disease burden, and respiratory function. Ultimately, this study will
generate extensive data regarding the clinical safety, pharmacokinetics, and change in body composition and
clinical function associated with combination therapy in a predefined FSHD population.

## Key facts

- **NIH application ID:** 9919651
- **Project number:** 5R01NS095813-04
- **Recipient organization:** UNIVERSITY OF ROCHESTER
- **Principal Investigator:** Chad Rydel Heatwole
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $471,791
- **Award type:** 5
- **Project period:** 2017-08-01 → 2023-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9919651

## Citation

> US National Institutes of Health, RePORTER application 9919651, STARFiSH: Study of Testosterone and rHGH in FSHD: A Proof-of-Concept Study (5R01NS095813-04). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/9919651. Licensed CC0.

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